Non-inferiority Trial Design at the Anti-Infectives Summit

Recently I had the pleasure of attending and presenting at the 3rd Annual Anti-Infectives Summit in Philadelphia. It was a good opportunity to interact and see presentations by people from medicinal chemistry, biochemistry, regulatory, and other backgrounds involved in drug discovery and research. I don’t deal much these days with the preclinical perspective, so it was a refreshing change of pace. I always appreciate the chance for cross-pollination of ideas with people from different areas.

There were a number of particularly good presentations, and one common theme that I saw was the borderline-inspiring enthusiasm by the researchers. You can always tell when someone truly loves what they do and is genuinely excited by their work – and I definitely saw that. Notables included Ying Zhang, MD, PhD, from Johns Hopkins, who opened my eyes in his presentation on issues with persister bacteria and Jennifer Schranz, MD from Cempra, who gave a great overview on the FDA’s expectations for clinical trials. She took what could have been a dry topic and infused it with energy and pace – I wish I could poach her to guest lecture in one of our drug information courses on this topic.

Something else that came out of the presentations was the increased emphasis on PROs (patient reported outcomes) in clinical trials. Considering that the immediate past-director of the NIH (Elias Zerhouni) made a statement about the increasing importance of the participatory medicine model…I think it was just a question of “when”, not “if”. That point spurred several interesting chats about PROs, ePatients, Phase IV, and the development of Pharmacovigilance 2.0.

Overall it was a very positive experience. I enjoyed some science refereshers, regulatory navigation tips, and insight into the future of anti-infective drug development.

@kevinclauson

Edit: I used an audience response system (aka clickers) in my presentation and included those slides here. However, I did not use the actual attendees’ responses in the slide deck I uploaded.

Superiority, Equivalence, and Non-inferiority Trial Design Lecture – Web 2.0 Style

A couple of weeks ago I read a very thoughtful post on one of my favorite blogs (authored by @laikas).  One reason I enjoy Jacqueline’s blog is that it contains evaluation (or at minimum consideration) of the information and literature that its posts are written about…rather than simply repackaging it without any context.  I do believe there is also clear value for blog posts that primarily serve current awareness, etc.  However, when a blog is augmented with the occasional reflective or methodical examination, it is elevated to a different level IMHO.  It’s somewhat akin to the difference in nominations for the medGadget Awards versus the Research Blogging Awards (chapeau tip to @DrVal).  The information found in both award contenders is valuable, but serves different purposes.     

In any event, the post about randomized, controlled trials (RCTs) and evidence based medicine (EBM) on Laika’s MedLibLog made me reconsider a lecture I had just finished putting together on trial design for a Drug Literature Evaluation course.  In that course, there are several lectures on different types of trial design as well as separate ones about EBM and clinical practice guidelines (CPGs) –  but they are all probably a little too insular.  Each lecture may be a little too focused on the technical aspects, and guilty of ignoring how each fits into the grand scheme of things.

The Point Of All This

When I first prepared my lecture on trial design, I think I was too preoccupied with conveying the importance of fundamentals to the students.  Reading the aforementioned post made me re-think my approach a bit and prompted me to revise the lecture/slides to incorporate a little more integration and application.  To that end, I posted them on Slideshare.  For me, this sequence of events is a perfect example of the underlying concepts of Web 2.0 in action…and that’s pretty cool.

@kevinclauson

P.S. I used the Guided Notes approach (which is why it looks like there is a preponderance of underlined text) and an audience response system in this lecture.